Science

Britain approves ‘world’s most expensive’ drug costing ₹18 crore

Prashasti Awasthi Mumbai | Updated on March 09, 2021

Zolgensma is used to cure Spinal Muscular Atrophy

The UK’s National Health Service (NHS) has approved the world’s most expensive drug that can prevent a rare genetic disorder, as per media reports.

The drug, called Zolgensma and manufactured by Novartis Gene Therapies, has been approved by Britain. The drug costs ₹18 crore (£1.79 million) per dose, according to the official statement NHS England.

The drug is used to cure Spinal Muscular Atrophy (SMA). SMA is rare and can be a fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.

This drug can treat the genetic disorder, especially prevalent among babies and young children that makes their life expectancy only two years.

Zolgensma can help babies breathe without a ventilator, sit up on their own, and crawl and walk after a single infusion treatment, as per previous studies.

NHS noted that the treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1.

NHS explained that the active ingredient onasemnogene abeparvovec present in the drug passes into the nerves and restores the gene. This then produces proteins essential for nerve function and controlling muscle movement.

The latest data provided by the NHS revealed that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and improve life expectancy.

“This deal is a life-changer for youngsters with this cruel disease and for their families,” said NHS England chief executive Sir Simon Stevens.

“Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers,” Stevens stated.

Published on March 09, 2021

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